Use of Ultrase® MT12 in Young Cystic Fibrosis Children (CF)

NCT00880100 | Completed Phase 3 Results DMC

• 1 other identifier: UMT12CF08-01
• Study Type: interventional
• Target: 49
• Locations: 1 country
• Sites: 15

Brief Summary

Multicenter, explorative, phase IIIb, open-label study to assess the efficacy and safety of Ultrase® MT12, in the control of steatorrhea and clinical signs and symptoms of malabsorption in CF children with pancreatic insufficiency (PI). This study is sponsored by Aptalis Pharma (formerly Axcan).

Conditions

Cystic Fibrosis; Pancreatic Insufficiency

Keywords

Steatorrhea; Malabsorption of fat; Pancreatic enzymes; Abdominal pain; Greasy stools

Outcome Measures

Primary Outcomes (1)

• Percentage of Patients With Control of Steatorrhea

  Control of steatorrhea was defined as a less than 30 percent (%) of fat in stools as measured by nuclear magnetic resonance (NMR) spectroscopy in all stool samples which are collected at baseline phase (usual pancreatic enzymes) during which the patients were on their prescribed pancreatic enzyme product (PEP) and during the 5-day collection period of the treatment phase during which the PEP was replaced with Ultrase MT12.

  A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)

Secondary Outcomes (4)

• Percentage of Patients With Normal Stool Frequency

  A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)

• Percentage of Stools With Normal Consistency

  A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)

• Percentage of Stools With Abnormal Characteristics

  A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)

• Mean Number of Days Without Abdominal Complaints

  A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)

Other Outcomes (2)

• Total Weight of Stools

  A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)

• Percentage of Days With Abdominal Pain and Excessive Flatulence

  A period of 19 to 24 days, from Baseline (Visit 2) to Day 15 to19 of Treatment Phase (Visit 3)

Study Arms (1)

Ultrase® MT12

EXPERIMENTAL

Drug: Ultrase® MT12

Interventions

Ultrase® MT12 DRUG

Ultrase® MT12 capsules will be given orally daily based on investigator's discretion to a maximum dose of 2,500 lipase units per kilogram (kg) body weight per meal or snack for 19 to 24 days during the treatment phase. Total maximum dose not to exceed 10,000 lipase units/kg/day.

Ultrase® MT12

Eligibility Criteria

• Age: 2 Years - 6 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Male or female patients aged 2 to 6 years inclusively
• Patients with current diagnosis of CF based on one or more typical clinical features of CF or a sibling with CF or a positive newborn screening and at least either with sweat chloride test greater than or equal to 60 millimoles/liter (mmol/L) by quantitative pilocarpine iontophoresis on two separate occasions or two identifiable CF-causing mutations
• Patients with presence of PI as demonstrated by fecal elastase (FE-1) less than 100 microgram/gram (mcg/g) of stools (performed by ScheBo test) and requiring pancreatic enzyme supplementation
• Patients who are able to eat a high-fat diet calculated at a value between 2g to 4g fat/kg of body weight per day during the whole study and having a current adequate nutritional status based on the body mass index (BMI) greater than or equal to fifth percentile
• Patients receiving current treatment of PI with pancreatic enzymes
• The parent or legal guardian signed informed consent form (ICF) and is mentally able to understand and comply with the study procedures

You may not qualify if:

• Patients currently receiving or received an Ultrase® MT product (MT12, MT18, MT20) for PI in the last 30 days
• Patients having known contraindication, sensitivity or hypersensitivity to Ultrase® or to any porcine protein
• Patients with presence of a medical condition known to increase fecal fat loss or that could compromise study results or the study patient safety
• Patients with current diagnosis or history of complete distal intestinal obstruction syndrome (DIOS) in the past 6 months or who had 2 or more episodes of incomplete DIOS in the past year
• Patients with use of any prohibited medication or product at study entry and during the course of the study
• Patients with chronic use of narcotics
• Patients with use of bowel stimulants and/or laxatives more than once a week
• Patients with presence of acute pancreatitis or exacerbation of chronic pancreatic disease
• Patients with presence of an acute infection that needed to be treated with oral or intravenous (IV) broad-spectrum antibiotics
• Patients having history of significant bowel resection; small bowel resection for meconium ileus at birth and appendectomy were accepted. Patients with Presence of dysmotility disorders
• Patients with presence of chronic or severe abdominal pain
• Patients unable to comply with diet requirement
• Patients receiving enteral tube feeding overnight at study entry or who will need to receive enteral tube feeding overnight during the course of the study
• Patients with history of or a current diagnosis of clinically significant portal hypertension
• Patients with presence of poorly controlled diabetes according to the Investigator's clinical judgment

Sponsors & Collaborators

• Forest Laboratories: lead

Study Sites (15)

The Children's Hospital

Aurora, Colorado, 80045, United States

Location

University of Michigan Health System Cystic Fibrosis Center

Ann Arbor, Michigan, 48109-0212, United States

Location

Helen DeVos Children's Hospital-Spectrum Health Research Department

Grand Rapids, Michigan, 40503, United States

Location

SUNY Upstate Medical University

Syracuse, New York, 13203, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Rainbow Babies and Children's Hospital - Cystic Fibrosis Center

Cleveland, Ohio, 44106, United States

Location

Children's Medical Center of Dayton

Dayton, Ohio, 45404, United States

Location

University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, 73104, United States

Location

Respiratory Diseases of Children and Adolescents

Oklahoma City, Oklahoma, 73112, United States

Location

Pennsylvania State University and the Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15213, United States

Location

Sanford Children's Specialty Clinic

Sioux Falls, South Dakota, 57117-5039, United States

Location

University of Utah

Salt Lake City, Utah, 84108, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23298, United States

Location

UW Hospital and Clinics

Madison, Wisconsin, 53792, United States

Location

MeSH Terms

Conditions

Cystic Fibrosis; Exocrine Pancreatic Insufficiency; Steatorrhea; Abdominal Pain

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Malabsorption Syndromes; Intestinal Diseases; Gastrointestinal Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Pain; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Signs and Symptoms, Digestive

Results Point of Contact

• Title: Robert Winkler, MD, VP, Clinical Development and Operations
• Organization: Aptalis Pharma US, Inc.

1- 800- 472- 263

Study Officials

• Aptalis Medical Information

  Forest Laboratories

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 9, 2009
• First Posted: April 13, 2009
• Study Start: April 1, 2009
• Primary Completion: November 1, 2009
• Study Completion: November 1, 2009
• Last Updated: March 16, 2017
• Results First Posted: February 19, 2015

Record last verified: 2017-02

Locations

US (15)